The Hematopoietic Cell Transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases

被引:52
作者
Thakar, Monica S. [1 ]
Broglie, Larisa [1 ]
Logan, Brent [2 ]
Artz, Andrew [3 ]
Bunin, Nancy [4 ]
Burroughs, Lauri M. [5 ,6 ]
Fretham, Caitrin [7 ]
Jacobsohn, David A. [8 ,9 ]
Loren, Alison W. [10 ]
Kurtzberg, Joanne [11 ]
Martinez, Caridad A. [12 ,13 ]
Mineishi, Shin [14 ]
Nelson, Adam S. [15 ]
Woolfrey, Ann [5 ,6 ]
Pasquini, Marcelo C. [16 ,17 ]
Sorror, Mohamed L. [5 ,18 ]
机构
[1] Med Coll Wisconsin, Div Pediat Hematol Oncol & Blood & Marrow Transpl, Dept Pediat, Milwaukee, WI 53226 USA
[2] Med Coll Wisconsin, Div Biostat, Milwaukee, WI 53226 USA
[3] Univ Chicago, Dept Med, Sect Hematol Oncol, 5841 S Maryland Ave, Chicago, IL 60637 USA
[4] Childrens Hosp Philadelphia, Dept Pediat, Div Oncol, Cellular Therapy & Transplant Sect, Philadelphia, PA 19104 USA
[5] Fred Hutchinson Canc Res Ctr, Clin Res Div, 1124 Columbia St, Seattle, WA 98104 USA
[6] Univ Washington, Dept Pediat, Div Pediat Hematol Oncol & Blood & Marrow Transpl, Seattle, WA 98195 USA
[7] Natl Marrow Donor Program, Ctr Int Blood & Marrow Transplant Res, Div Biostat, Minneapolis, MN USA
[8] Childrens Natl Hlth Syst, Dept Pediat, Div Blood & Marrow Transplantat, Washington, DC USA
[9] George Washington Univ, Washington, DC USA
[10] Univ Penn, Dept Med, Div Hematol Oncol, Philadelphia, PA 19104 USA
[11] Duke Univ, Med Ctr, Dept Pediat, Div Pediat Blood & Marrow Transplant, Durham, NC 27710 USA
[12] Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX 77030 USA
[13] Texas Childrens Hosp, Houston, TX 77030 USA
[14] Penn State Hershey Med Ctr, Div Hematol & Oncol, Dept Med, Blood & Marrow Transplant Program, Hershey, PA USA
[15] Cincinnati Childrens Hosp, Dept Pediat, Div Bone Marrow Transplant & Immune Deficiency, Cincinnati, OH USA
[16] Ctr Int Blood & Marrow Transplant Res, Milwaukee, WI USA
[17] Med Coll Wisconsin, Dept Med, Div Hematol & Oncol, Milwaukee, WI 53226 USA
[18] Univ Washington, Dept Med, Div Oncol, Seattle, WA 98195 USA
基金
美国国家卫生研究院;
关键词
SEVERE COMBINED IMMUNODEFICIENCY; MARROW-TRANSPLANTATION; REDUCED-INTENSITY; RISK-ASSESSMENT; VALIDATION; OUTCOMES; MORTALITY; MORBIDITY; BLOOD; AGE;
D O I
10.1182/blood-2018-09-876284
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Despite improvements, mortality after allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases remains a significant problem. We evaluated whether pre-HCT conditions defined by the HCT Comorbidity Index (HCT-CI) predict probability of post-transplant survival. Using the Center for International Blood and Marrow Transplant Research database, we identified 4083 patients with nonmalignant diseases transplanted between 2007 and 2014. Primary outcome was overall survival (OS) using the Kaplan-Meier method. Hazard ratios (HRs) were estimated by multivariable Cox regression models. Increasing HCT-CI scores translated to decreased 2-year OS of 82.7%, 80.3%, 74%, and 55.8% for patients with HCT-CI scores of 0, 1 to 2, 3 to 4, and >= 5, respectively, regardless of conditioning intensity. HCT-CI scores of 1 to 2 did not differ relative to scores of 0 (HR, 1.12 [95% CI, 0.93-1.34]), but HCT-CI of 3 to 4 and >= 5 posed significantly greater risks of mortality (HR, 1.33 [95% CI, 1.09-1.63]; and HR, 2.31 [95% CI, 1.79-2.96], respectively). The effect of HCT-CI differed by disease indication. Patients with acquired aplastic anemia, primary immune deficiencies, and congenital bone marrow failure syndromes with scores >= 3 had increased risk of death after HCT. However, higher HCT-CI scores among hemoglobinopathy patients did not increase mortality risk. In conclusion, this is the largest study to date reporting on patients with nonmalignant diseases demonstrating HCT-CI scores >= 3 that had inferior survival after HCT, except for patients with hemoglobinopathies. Our findings suggest that using the HCT-CI score, in addition to disease-specific factors, could be useful when developing treatment plans for nonmalignant diseases.
引用
收藏
页码:754 / 762
页数:9
相关论文
共 36 条
[1]   Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines [J].
Aldenhoven, Mieke ;
Jones, Simon A. ;
Bonney, Denise ;
Borrill, Roisin E. ;
Coussons, Mary ;
Mercer, Jean ;
Bierings, Marc B. ;
Versluys, Birgitta ;
van Hasselt, Peter M. ;
Wijburg, Frits A. ;
van der Ploeg, Ans T. ;
Wynn, Robert F. ;
Boelens, Jaap Jan .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2015, 21 (06) :1106-1109
[2]   Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis [J].
Bacigalupo, Andrea ;
Socie, Gerard ;
Hamladji, Rose Marie ;
Aljurf, Mahmoud ;
Maschan, Alexei ;
Kyrcz-Krzemien, Slawomira ;
Chybicka, Alicja ;
Sengelov, Henrik ;
Unal, Ali ;
Beelen, Dietrich ;
Locasciulli, Anna ;
Dufour, Carlo ;
Passweg, Jakob R. ;
Oneto, Rosi ;
Signori, Alessio ;
Marsh, Judith C. W. .
HAEMATOLOGICA, 2015, 100 (05) :696-702
[3]   Defining the Intensity of Conditioning Regimens: Working Definitions [J].
Bacigalupo, Andrea ;
Ballen, Karen ;
Rizzo, Doug ;
Giralt, Sergio ;
Lazarus, Hillard ;
Ho, Vincent ;
Apperley, Jane ;
Slavin, Shimon ;
Pasquini, Marcelo ;
Sandmaier, Brenda M. ;
Barrett, John ;
Blaise, Didier ;
Lowski, Robert ;
Horowitz, Mary .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2009, 15 (12) :1628-1633
[4]   Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future [J].
Bhatia, M. ;
Walters, M. C. .
BONE MARROW TRANSPLANTATION, 2008, 41 (02) :109-117
[5]   The Burden of Comorbidity and Complexity in Sarcoidosis: Impact of Associated Chronic Diseases [J].
Brito-Zeron, Pilar ;
Acar-Denizli, Nihan ;
Siso-Almirall, Antoni ;
Bosch, Xavier ;
Hernandez, Fernanda ;
Vilanova, Sergi ;
Villalta, Mireia ;
Kostov, Belchin ;
Paradela, Marina ;
Sanchez, Marcelo ;
Ramirez, Jose ;
Muxi, Africa ;
Berruezo, Antonio ;
Galceran-Chaves, Celeste ;
Xaubet, Antoni ;
Agusti, Carles ;
Sellares, Jacobo ;
Ramos-Casals, Manuel .
LUNG, 2018, 196 (02) :239-248
[6]   VALIDATION OF A COMBINED COMORBIDITY INDEX [J].
CHARLSON, M ;
SZATROWSKI, TP ;
PETERSON, J ;
GOLD, J .
JOURNAL OF CLINICAL EPIDEMIOLOGY, 1994, 47 (11) :1245-1251
[7]  
COX DR, 1972, J R STAT SOC B, V34, P187
[8]   The Natural History of Children with Severe Combined Immunodeficiency: Baseline Features of the First Fifty Patients of the Primary Immune Deficiency Treatment Consortium Prospective Study 6901 [J].
Dvorak, Christopher C. ;
Cowan, Morton J. ;
Logan, Brent R. ;
Notarangelo, Luigi D. ;
Griffith, Linda M. ;
Puck, Jennifer M. ;
Kohn, Donald B. ;
Shearer, William T. ;
O'Reilly, Richard J. ;
Fleisher, Thomas A. ;
Pai, Sung-Yun ;
Hanson, I. Celine ;
Pulsipher, Michael A. ;
Fuleihan, Ramsay ;
Filipovich, Alexandra ;
Goldman, Frederick ;
Kapoor, Neena ;
Small, Trudy ;
Smith, Angela ;
Chan, Ka-Wah ;
Cuvelier, Geoff ;
Heimall, Jennifer ;
Knutsen, Alan ;
Loechelt, Brett ;
Moore, Theodore ;
Buckley, Rebecca H. .
JOURNAL OF CLINICAL IMMUNOLOGY, 2013, 33 (07) :1156-1164
[9]   Up-to-date tools for risk assessment before allogeneic hematopoietic cell transplantation [J].
Elsawy, M. ;
Sorror, M. L. .
BONE MARROW TRANSPLANTATION, 2016, 51 (10) :1283-1300
[10]   Multi-centre validation of the prognostic value of the haematopoietic cell transplantation- specific comorbidity index among recipient of allogeneic haematopoietic cell transplantation [J].
ElSawy, Mahmoud ;
Storer, Barry E. ;
Pulsipher, Michael A. ;
Maziarz, Richard T. ;
Bhatia, Smita ;
Maris, Michael B. ;
Syrjala, Karen L. ;
Martin, Paul J. ;
Maloney, David G. ;
Sandmaier, Brenda M. ;
Storb, Rainer ;
Sorror, Mohamed L. .
BRITISH JOURNAL OF HAEMATOLOGY, 2015, 170 (04) :574-583